RARE Daily

QurAlis and Unlearn Enter Collaboration to Use Digital Twins to Improve ALS Clinical Trials

June 27, 2023

Rare Daily Staff

QurAlis has entered into a collaboration with the AI company Unlearn to optimize QurAlis’ clinical program in the rare, neurodegenerative condition ALS using Unlearn’s advanced generative artificial intelligence technology.

Unlearn develops digital twins of clinical trial patients that are predictions of individual health outcomes under the control treatment over time. Digital twins are employed in randomized controlled trials to run more efficient trials that produce regulatory-suitable evidence.

The collaboration aims to reduce variability and increase the study power in QurAlis’ clinical trials for QRL-201 and QRL-101, the company’s lead product candidates in ALS. Unlearn’s machine learning models are trained on existing clinical data. After validation, they are used to generate digital twins from baseline data for each patient enrolled in a randomized control trial or TwinRCT, regardless of their randomization assignment. Prognostic scores derived from digital twins are incorporated into the trial’s primary analysis to precisely estimate treatment effects and control for false positives.

“By using machine learning to leverage the wealth of existing patient data from completed clinical trials, our technology significantly shortens typical timelines by months while generating evidence suitable for supporting regulatory decisions,” said Charles Fisher, founder and CEO of Unlearn. “The AI technology we’re developing today will revolutionize the future of clinical research.”

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive disease impacting nerve cells in the brain and spinal cord. ALS breaks down nerve cells, reducing muscle function and causing loss of muscle control. ALS can be traced to mutations in more than 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Its average life expectancy is three years, and there is currently no cure for the disease.

QRL-201 is a first-in-class experimental therapy that seeks to restore STATHMIN-2 (STMN2) expression in ALS patients. STATHMIN-2 is a well-validated protein important for neural repair and axonal stability, the expression of which is significantly decreased in nearly all ALS patients. QRL-201 rescues STMN2 loss of function in QurAlis ALS patient-derived motor neuron disease models.

In December 2022, QurAlis announced the company-initiated dosing of QRL-101 in a first-in-human phase 1 clinical trial. QRL-101 is a first-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS.

Photo: Charles Fisher, founder and CEO of Unlearn

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